Trial Outcomes & Findings for Study of Efficacy and Safety of QAW039 When Added to Standard-of-care Asthma Therapy in Patients With Uncontrolled Asthma (NCT NCT03215758)

NCT ID: NCT03215758

Last Updated: 2026-01-13

Results Overview

Forced Expiratory Volume in one second (FEV1) is calculated as the volume of air forcibly exhaled in one second as measured by a spirometer. Baseline is defined as the last available FEV1 measurement taken prior to the first dose of randomized study drug.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

675 participants

Primary outcome timeframe

Week 12

Results posted on

2026-01-13

Participant Flow

Participants were recruited from centers in Argentina (20), Germany (12), Hungary (4), Mexico (2), Philippines (4), Slovakia (7), South Africa (5), Turkey (5), United States (29)

Participant milestones

Participant milestones
Measure
QAW039
QAW039 once daily
Placebo
Placebo once daily
Overall Study
STARTED
339
336
Overall Study
COMPLETED
334
328
Overall Study
NOT COMPLETED
5
8

Reasons for withdrawal

Reasons for withdrawal
Measure
QAW039
QAW039 once daily
Placebo
Placebo once daily
Overall Study
Technical Problems
1
0
Overall Study
Protocol deviation
0
1
Overall Study
Physician Decision
0
1
Overall Study
Death
0
1
Overall Study
Adverse Event
1
2
Overall Study
Subject/Guardian Decision
3
3

Baseline Characteristics

Study of Efficacy and Safety of QAW039 When Added to Standard-of-care Asthma Therapy in Patients With Uncontrolled Asthma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
QAW039
n=339 Participants
QAW039 once daily
Placebo
n=336 Participants
Placebo once daily
Total
n=675 Participants
Total of all reporting groups
Age, Continuous
48.1 Years
STANDARD_DEVIATION 15.15 • n=210 Participants
47.7 Years
STANDARD_DEVIATION 15.40 • n=19 Participants
47.9 Years
STANDARD_DEVIATION 15.26 • n=123 Participants
Sex: Female, Male
Female
217 Participants
n=210 Participants
216 Participants
n=19 Participants
433 Participants
n=123 Participants
Sex: Female, Male
Male
122 Participants
n=210 Participants
120 Participants
n=19 Participants
242 Participants
n=123 Participants
Race/Ethnicity, Customized
Caucasian
274 Participants
n=210 Participants
281 Participants
n=19 Participants
555 Participants
n=123 Participants
Race/Ethnicity, Customized
Black
18 Participants
n=210 Participants
12 Participants
n=19 Participants
30 Participants
n=123 Participants
Race/Ethnicity, Customized
Asian
39 Participants
n=210 Participants
34 Participants
n=19 Participants
73 Participants
n=123 Participants
Race/Ethnicity, Customized
Native American
1 Participants
n=210 Participants
0 Participants
n=19 Participants
1 Participants
n=123 Participants
Race/Ethnicity, Customized
Pacific Islander
2 Participants
n=210 Participants
0 Participants
n=19 Participants
2 Participants
n=123 Participants
Race/Ethnicity, Customized
Unknown
0 Participants
n=210 Participants
3 Participants
n=19 Participants
3 Participants
n=123 Participants
Race/Ethnicity, Customized
Other
5 Participants
n=210 Participants
6 Participants
n=19 Participants
11 Participants
n=123 Participants

PRIMARY outcome

Timeframe: Week 12

Population: Full analysis set (FAS): all randomized patients who received at least one dose of study medication. Patients in the FAS were analyzed according to the treatment they were assigned to at randomization.

Forced Expiratory Volume in one second (FEV1) is calculated as the volume of air forcibly exhaled in one second as measured by a spirometer. Baseline is defined as the last available FEV1 measurement taken prior to the first dose of randomized study drug.

Outcome measures

Outcome measures
Measure
QAW039
n=339 Participants
QAW039 once daily
Placebo
n=336 Participants
Placebo once daily
Change From Baseline in Pre-dose FEV1 at Week 12
0.112 Liters
Standard Error 0.0167
0.071 Liters
Standard Error 0.0169

SECONDARY outcome

Timeframe: 12 weeks

Population: Full analysis set (FAS): all randomized patients who received at least one dose of study medication. Patients in the FAS were analyzed according to the treatment they were assigned to at randomization.

Daytime asthma symptoms are evaluated through four questions and each of them will be rated on a scale of 0 to 6. Higher scores indicate more severe asthma-related symptoms. A mean score will be calculated for the responses to 4 questions.

Outcome measures

Outcome measures
Measure
QAW039
n=339 Participants
QAW039 once daily
Placebo
n=336 Participants
Placebo once daily
Change From Baseline in Daytime Asthma Symptom Score
-0.56 Score
Standard Error 0.036
-0.51 Score
Standard Error 0.037

SECONDARY outcome

Timeframe: 12 weeks

Population: Full analysis set (FAS): all randomized patients who received at least one dose of study medication. Patients in the FAS were analyzed according to the treatment they were assigned to at randomization.

Daily use of SABA (the number of rescue medication puffs taken in the previous 12 hours) was recorded using a patient electronic diary (referred to as eDiary or eDiary/ePEF). Patients were instructed to routinely complete the patient diary twice daily - at the same time each morning and each evening, approximately 12 hours apart.

Outcome measures

Outcome measures
Measure
QAW039
n=339 Participants
QAW039 once daily
Placebo
n=336 Participants
Placebo once daily
Change From Baseline in Daily Use of SABA
-1.11 Number of puffs
Standard Error 0.075
-1.02 Number of puffs
Standard Error 0.076

SECONDARY outcome

Timeframe: Week 12

Population: Full analysis set (FAS): all randomized patients who received at least one dose of study medication. Patients in the FAS were analyzed according to the treatment they were assigned to at randomization.

AQLQ is a 32-item instrument administered as a self-assessment. AQLQ+12 is a modified version of AQLQ developed to measure functional impairments of participants aged 12-70 years. It is divided into 4 domains: activity limitation, symptoms, emotional function, and environmental stimuli. Participants were asked to recall their experiences during the last 2 weeks and respond to each question on a 7-point scale (1=severe impairment, 7=no impairment), where higher scores indicated "better quality of life." Overall AQLQ+12 score is the mean of all 32 responses.

Outcome measures

Outcome measures
Measure
QAW039
n=339 Participants
QAW039 once daily
Placebo
n=336 Participants
Placebo once daily
Change From Baseline in Asthma Quality of Life (AQLQ+12) Score
0.91 units on a scale
Standard Error 0.048
0.89 units on a scale
Standard Error 0.049

Adverse Events

QAW039 150 mg

Serious events: 1 serious events
Other events: 94 other events
Deaths: 0 deaths

Placebo

Serious events: 5 serious events
Other events: 102 other events
Deaths: 1 deaths

Serious adverse events

Serious adverse events
Measure
QAW039 150 mg
n=339 participants at risk
QAW039 150 mg
Placebo
n=336 participants at risk
Placebo
Infections and infestations
Upper respiratory tract infection bacterial
0.00%
0/339 • After signing informed consent to 30 days after last dose
0.30%
1/336 • After signing informed consent to 30 days after last dose
Investigations
Electrocardiogram T wave inversion
0.00%
0/339 • After signing informed consent to 30 days after last dose
0.30%
1/336 • After signing informed consent to 30 days after last dose
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Astrocytoma
0.00%
0/339 • After signing informed consent to 30 days after last dose
0.30%
1/336 • After signing informed consent to 30 days after last dose
Respiratory, thoracic and mediastinal disorders
Asthma
0.29%
1/339 • After signing informed consent to 30 days after last dose
0.60%
2/336 • After signing informed consent to 30 days after last dose
Respiratory, thoracic and mediastinal disorders
Pulmonary embolism
0.00%
0/339 • After signing informed consent to 30 days after last dose
0.30%
1/336 • After signing informed consent to 30 days after last dose

Other adverse events

Other adverse events
Measure
QAW039 150 mg
n=339 participants at risk
QAW039 150 mg
Placebo
n=336 participants at risk
Placebo
Gastrointestinal disorders
Diarrhoea
0.59%
2/339 • After signing informed consent to 30 days after last dose
1.2%
4/336 • After signing informed consent to 30 days after last dose
General disorders
Fatigue
0.59%
2/339 • After signing informed consent to 30 days after last dose
1.2%
4/336 • After signing informed consent to 30 days after last dose
Infections and infestations
Acute sinusitis
1.2%
4/339 • After signing informed consent to 30 days after last dose
0.30%
1/336 • After signing informed consent to 30 days after last dose
Infections and infestations
Bronchitis
3.5%
12/339 • After signing informed consent to 30 days after last dose
3.0%
10/336 • After signing informed consent to 30 days after last dose
Infections and infestations
Nasopharyngitis
3.8%
13/339 • After signing informed consent to 30 days after last dose
3.6%
12/336 • After signing informed consent to 30 days after last dose
Infections and infestations
Rhinitis
0.29%
1/339 • After signing informed consent to 30 days after last dose
1.5%
5/336 • After signing informed consent to 30 days after last dose
Infections and infestations
Upper respiratory tract infection
1.8%
6/339 • After signing informed consent to 30 days after last dose
3.0%
10/336 • After signing informed consent to 30 days after last dose
Infections and infestations
Upper respiratory tract infection bacterial
1.5%
5/339 • After signing informed consent to 30 days after last dose
1.5%
5/336 • After signing informed consent to 30 days after last dose
Infections and infestations
Viral upper respiratory tract infection
2.1%
7/339 • After signing informed consent to 30 days after last dose
1.8%
6/336 • After signing informed consent to 30 days after last dose
Investigations
Blood creatine phosphokinase increased
0.59%
2/339 • After signing informed consent to 30 days after last dose
1.5%
5/336 • After signing informed consent to 30 days after last dose
Musculoskeletal and connective tissue disorders
Arthralgia
0.29%
1/339 • After signing informed consent to 30 days after last dose
1.2%
4/336 • After signing informed consent to 30 days after last dose
Musculoskeletal and connective tissue disorders
Back pain
1.2%
4/339 • After signing informed consent to 30 days after last dose
1.2%
4/336 • After signing informed consent to 30 days after last dose
Nervous system disorders
Headache
1.8%
6/339 • After signing informed consent to 30 days after last dose
2.1%
7/336 • After signing informed consent to 30 days after last dose
Respiratory, thoracic and mediastinal disorders
Asthma
12.7%
43/339 • After signing informed consent to 30 days after last dose
13.4%
45/336 • After signing informed consent to 30 days after last dose
Respiratory, thoracic and mediastinal disorders
Cough
0.59%
2/339 • After signing informed consent to 30 days after last dose
1.5%
5/336 • After signing informed consent to 30 days after last dose
Respiratory, thoracic and mediastinal disorders
Nasal congestion
0.00%
0/339 • After signing informed consent to 30 days after last dose
1.2%
4/336 • After signing informed consent to 30 days after last dose
Respiratory, thoracic and mediastinal disorders
Rhinitis allergic
3.5%
12/339 • After signing informed consent to 30 days after last dose
1.8%
6/336 • After signing informed consent to 30 days after last dose
Vascular disorders
Hypertension
0.29%
1/339 • After signing informed consent to 30 days after last dose
1.5%
5/336 • After signing informed consent to 30 days after last dose

Additional Information

Study Director

Novartis Pharmaceutical

Phone: 862-778-8300

Results disclosure agreements

  • Principal investigator is a sponsor employee The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (ie, data from all sites) in the clinical trial.
  • Publication restrictions are in place

Restriction type: OTHER